首页 > 最新文献

Value in Health最新文献

英文 中文
Development of a Health-State Classification System for the Pediatric Quality-of-Life Inventory Version 4.0 Generic Core Scales for Preference-Based Valuation in Australia. 为 PedsQL™ 4.0 通用核心量表开发健康状况分类系统,用于在澳大利亚进行基于偏好的估值。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-09-27 DOI: 10.1016/j.jval.2024.08.005
Joseph Kwon, Rakhee Raghunandan, Son Hong Nghiem, Kirsten Howard, Emily Lancsar, Elisabeth Huynh, Martin Howell, Stavros Petrou, Sarah Smith

Objectives: Pediatric Quality-of-Life Inventory Version 4.0 Generic Core Scales (PedsQL GCS), comprising 23 items covering 4 subscales (physical, emotional, social, and school functioning), is a widely applied generic measure of childhood health-related quality of life but does not provide health utilities for cost-effectiveness-based decision making. This study aimed to develop a reduced item version of PedsQL GCS amenable to health utility derivation in Australia.

Methods: Data sources were 2 cohorts of the Longitudinal Study of Australian Children, including proxy responses for all PedsQL GCS versions (Toddlers, Young Children, Children, and Teens), and the CheckPoint sample containing child self-report to the Children version. Three analytic samples were CheckPoint sample (n = 1874); Mallinson sample containing 1 measurement per child from one of the Young Children, Children, or Teens versions (n = 7855); and Toddlers sample (n = 7401). Exploratory and confirmatory factor analyses assessed dimensionality. Psychometric analyses used Rasch and classical criteria on 3 randomly selected subsamples (n = 500) per sample. Item selection prioritized psychometric performance in the CheckPoint sample, also considering performance in other samples and conceptual content.

Results: Dimensionality assessments did not generate an alternative empirical structure for the measure, and psychometric analyses were conducted on the original 4 subscales. The selected items were: "Get aches and pains" for physical functioning; "Feel sad/blue" for emotional functioning; "Other kids not friends" for social functioning; and "Keeping up with school work" for school functioning.

Conclusions: The final 4-item set, pending further psychometric validation and valuation, can generate health utilities from the widely used PedsQL GCS to inform cost-effectiveness-based decision making.

目的:儿科生活质量量表(Pediatric Quality of Life InventoryTM Version 4.0 Generic Core Scales,简称 PedsQL GCS)由 23 个项目组成,涵盖四个分量表(身体、情绪、社交和学校功能),是一种广泛应用的儿童健康相关生活质量通用测量方法,但不能为基于成本效益的决策提供健康效用。本研究旨在开发一个可用于澳大利亚健康效用推导的 PedsQL GCS 简化项目版本:数据来源于《澳大利亚儿童纵向研究》的两个队列,包括所有 PedsQL GCS 版本(幼儿、幼儿、儿童、青少年)的代理回复,以及包含儿童版本自我报告的 CheckPoint 样本。三个分析样本分别是CheckPoint 样本(n=1,874)、Mallinson 样本(n=7,855)和幼儿样本(n=7,401)。探索性和确认性因素分析对维度进行了评估。心理测量分析对每个样本随机抽取的三个子样本(样本数=500)采用了 Rasch 和经典标准。项目选择优先考虑在 CheckPoint 样本中的心理测量表现,同时也考虑在其他样本中的表现和概念内容:维度评估没有为测量结果生成其他经验结构,因此对原有的四个子量表进行了心理测量分析。选定的项目有身体机能:"疼痛";情绪机能:"悲伤/忧郁";社会功能:"其他孩子不是朋友";学校功能:"跟上学校功课":最终的四项目集(有待进一步的心理计量验证和评估)可以从广泛使用的儿童生活质量量表 GCS 中生成健康效用,为基于成本效益的决策提供依据。
{"title":"Development of a Health-State Classification System for the Pediatric Quality-of-Life Inventory Version 4.0 Generic Core Scales for Preference-Based Valuation in Australia.","authors":"Joseph Kwon, Rakhee Raghunandan, Son Hong Nghiem, Kirsten Howard, Emily Lancsar, Elisabeth Huynh, Martin Howell, Stavros Petrou, Sarah Smith","doi":"10.1016/j.jval.2024.08.005","DOIUrl":"10.1016/j.jval.2024.08.005","url":null,"abstract":"<p><strong>Objectives: </strong>Pediatric Quality-of-Life Inventory Version 4.0 Generic Core Scales (PedsQL GCS), comprising 23 items covering 4 subscales (physical, emotional, social, and school functioning), is a widely applied generic measure of childhood health-related quality of life but does not provide health utilities for cost-effectiveness-based decision making. This study aimed to develop a reduced item version of PedsQL GCS amenable to health utility derivation in Australia.</p><p><strong>Methods: </strong>Data sources were 2 cohorts of the Longitudinal Study of Australian Children, including proxy responses for all PedsQL GCS versions (Toddlers, Young Children, Children, and Teens), and the CheckPoint sample containing child self-report to the Children version. Three analytic samples were CheckPoint sample (n = 1874); Mallinson sample containing 1 measurement per child from one of the Young Children, Children, or Teens versions (n = 7855); and Toddlers sample (n = 7401). Exploratory and confirmatory factor analyses assessed dimensionality. Psychometric analyses used Rasch and classical criteria on 3 randomly selected subsamples (n = 500) per sample. Item selection prioritized psychometric performance in the CheckPoint sample, also considering performance in other samples and conceptual content.</p><p><strong>Results: </strong>Dimensionality assessments did not generate an alternative empirical structure for the measure, and psychometric analyses were conducted on the original 4 subscales. The selected items were: \"Get aches and pains\" for physical functioning; \"Feel sad/blue\" for emotional functioning; \"Other kids not friends\" for social functioning; and \"Keeping up with school work\" for school functioning.</p><p><strong>Conclusions: </strong>The final 4-item set, pending further psychometric validation and valuation, can generate health utilities from the widely used PedsQL GCS to inform cost-effectiveness-based decision making.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354770","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Economic Evaluation of Fasting Mimicking Diet vs Standard Care in Diabetic Patients on Dual or Triple Medications at Baseline in the United States: A Cost-Utility Analysis. 对美国基线服用双重或三重药物的糖尿病患者进行空腹模拟饮食与标准护理的经济评估:成本效用分析。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-09-27 DOI: 10.1016/j.jval.2024.08.003
Dany Habka, William C Hsu, Joseph Antoun

Objectives: According to most guidelines, dietary interventions are essential in the management of diabetes. Fasting has emerged as potential therapeutic regimes for diabetes. The proof-of-concept study and the fasting in diabetes treatment trial are the first to explore the clinical impact of the Fasting Mimicking Diet (FMD) in patients with type 2 diabetes mellitus. Their results showed that FMD cycles improve glycemic management and can be integrated into usual care complementary to current guidelines. This economic evaluation aims to assess the 10-year quality-of-life effects, cost implications, and cost-effectiveness of adding a 3-year FMD program to diabetes standard care in diabetic population on dual or triple medications at baseline from the perspective of the US payer.

Methods: We constructed a microsimulation model in TreeAge using a published US-specific diabetes model. The model was populated using FMD effectiveness outcomes and publicly available clinical and economic data associated with diabetes complications, use of diabetes medications, hypoglycemia incidence, direct medical costs in 2021 USD, quality of life, and mortality. All benefits were discounted by 3%.

Results: This cost-utility analysis showed that the FMD program was associated with 11.4% less diabetes complications, 67.2% less overall diabetes medication use, and 45.0% less hypoglycemia events over the 10-year simulation period. The program generated an additional effectiveness benefit of 0.211 quality-adjusted life year and net monetary benefit of 41 613 USD per simulated patient. Thus, the FMD program is cost saving.

Conclusions: These results indicate that the FMD program is a beneficial first-line strategy in T2DM management.

目的:根据大多数指南,饮食干预对糖尿病的治疗至关重要。禁食已成为糖尿病的潜在治疗方案。概念验证研究和禁食治疗糖尿病试验首次探讨了禁食模拟饮食(FMD™)对 T2DM 患者的临床影响。他们的研究结果表明,FMD™ 循环可改善血糖管理,并可作为现行指南的补充纳入常规护理中。本经济评估旨在从美国支付方的角度,评估在糖尿病标准治疗的基础上增加为期 3 年的 FMD™ 计划对糖尿病患者 10 年生活质量的影响、成本影响以及成本效益:我们使用已发布的美国糖尿病模型在 TreeAge 中构建了一个微观模拟模型。该模型使用 FMD™ 效果、与糖尿病并发症相关的公开临床和经济数据、糖尿病药物使用、低血糖发生率、2021 美元的直接医疗成本、生活质量和死亡率。所有收益均按 3% 的折扣计算:成本效用分析表明,在 10 年模拟期内,FMD™ 计划可使糖尿病并发症减少 11.4%,糖尿病药物总用量减少 67.2%,低血糖事件减少 45.0%。该计划为每位模拟患者带来了 0.211 QALY 的额外有效性收益和 41,613 美元的净货币收益。因此,FMD™ 计划可以节约成本:这些结果表明,FMD™ 计划是治疗 T2DM 的一线策略。
{"title":"Economic Evaluation of Fasting Mimicking Diet vs Standard Care in Diabetic Patients on Dual or Triple Medications at Baseline in the United States: A Cost-Utility Analysis.","authors":"Dany Habka, William C Hsu, Joseph Antoun","doi":"10.1016/j.jval.2024.08.003","DOIUrl":"10.1016/j.jval.2024.08.003","url":null,"abstract":"<p><strong>Objectives: </strong>According to most guidelines, dietary interventions are essential in the management of diabetes. Fasting has emerged as potential therapeutic regimes for diabetes. The proof-of-concept study and the fasting in diabetes treatment trial are the first to explore the clinical impact of the Fasting Mimicking Diet (FMD) in patients with type 2 diabetes mellitus. Their results showed that FMD cycles improve glycemic management and can be integrated into usual care complementary to current guidelines. This economic evaluation aims to assess the 10-year quality-of-life effects, cost implications, and cost-effectiveness of adding a 3-year FMD program to diabetes standard care in diabetic population on dual or triple medications at baseline from the perspective of the US payer.</p><p><strong>Methods: </strong>We constructed a microsimulation model in TreeAge using a published US-specific diabetes model. The model was populated using FMD effectiveness outcomes and publicly available clinical and economic data associated with diabetes complications, use of diabetes medications, hypoglycemia incidence, direct medical costs in 2021 USD, quality of life, and mortality. All benefits were discounted by 3%.</p><p><strong>Results: </strong>This cost-utility analysis showed that the FMD program was associated with 11.4% less diabetes complications, 67.2% less overall diabetes medication use, and 45.0% less hypoglycemia events over the 10-year simulation period. The program generated an additional effectiveness benefit of 0.211 quality-adjusted life year and net monetary benefit of 41 613 USD per simulated patient. Thus, the FMD program is cost saving.</p><p><strong>Conclusions: </strong>These results indicate that the FMD program is a beneficial first-line strategy in T2DM management.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142354726","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Analytical Methods for Comparing Uncontrolled Trials With External Controls From Real-World Data: A Systematic Literature Review and Comparison With European Regulatory and Health Technology Assessment Practice. 从真实世界数据中比较无对照试验与外部对照的分析方法:系统性文献综述及与欧洲监管和健康技术评估实践的比较。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-09-04 DOI: 10.1016/j.jval.2024.08.002
Milou A Hogervorst, Kanaka V Soman, Helga Gardarsdottir, Wim G Goettsch, Lourens T Bloem

Objectives: This study aimed to provide an overview of analytical methods in scientific literature for comparing uncontrolled medicine trials with external controls from individual patient data real-world data (IPD-RWD) and to compare these methods with recommendations made in guidelines from European regulatory and health technology assessment (HTA) organizations and with their evaluations described in assessment reports.

Methods: A systematic literature review (until March 1, 2023) in PubMed and Connected Papers was performed to identify analytical methods for comparing uncontrolled trials with external controls from IPD-RWD. These methods were compared descriptively with methods recommended in method guidelines and encountered in assessment reports of the European Medicines Agency (2015-2020) and 4 European HTA organizations (2015-2023).

Results: Thirty-four identified scientific articles described analytical methods for comparing uncontrolled trial data with IPD-RWD-based external controls. The various methods covered controlling for confounding and/or dependent censoring, correction for missing data, and analytical comparative modeling methods. Seven guidelines also focused on research design, RWD quality, and transparency aspects, and 4 of those recommended analytical methods for comparisons with IPD-RWD. The methods discussed in regulatory (n = 15) and HTA (n = 35) assessment reports were often based on aggregate data and lacked transparency owing to the few details provided.

Conclusions: Literature and guidelines suggest a methodological approach to comparing uncontrolled trials with external controls from IPD-RWD similar to target trial emulation, using state-of-the-art methods. External controls supporting regulatory and HTA decision making were rarely in line with this approach. Twelve recommendations are proposed to improve the quality and acceptability of these methods.

目的:概述科学文献中利用患者个人真实世界数据(IPD-RWD)将无对照药物试验与外部对照进行比较的分析方法。此外,还将这些方法与欧洲监管机构和卫生技术评估(HTA)机构在指南中提出的建议及其在评估报告中描述的评价进行比较:方法:在PubMed和Connected Papers上进行了一次系统的文献综述(截至2023年3月1日),以确定将IPD-RWD中的非对照试验与外部对照进行比较的分析方法。将这些方法与方法指南中推荐的方法以及欧洲药品管理局(2015-2020 年)和四个欧洲 HTA 组织(2015-2023 年)的评估报告中出现的方法进行了描述性比较:34 篇已确定的科学文章介绍了将非对照试验数据与基于 IPD-RWD 的外部对照进行比较的分析方法。各种方法涵盖了混杂控制和/或从属删减、缺失数据校正以及分析比较建模方法。七份指南还侧重于研究设计、RWD 质量和透明度方面,其中四份指南推荐了与 IPD-RWD 进行比较的分析方法。监管报告(n=15)和 HTA 评估报告(n=35)中讨论的方法通常基于综合数据,并且由于提供的细节较少而缺乏透明度:文献和指南建议采用最先进的方法,将非对照试验与IPD-RWD的外部对照进行比较,类似于目标试验模拟。支持监管和 HTA 决策的外部对照很少符合这种方法。为提高这些方法的质量和可接受性,我们提出了 12 项建议。
{"title":"Analytical Methods for Comparing Uncontrolled Trials With External Controls From Real-World Data: A Systematic Literature Review and Comparison With European Regulatory and Health Technology Assessment Practice.","authors":"Milou A Hogervorst, Kanaka V Soman, Helga Gardarsdottir, Wim G Goettsch, Lourens T Bloem","doi":"10.1016/j.jval.2024.08.002","DOIUrl":"10.1016/j.jval.2024.08.002","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to provide an overview of analytical methods in scientific literature for comparing uncontrolled medicine trials with external controls from individual patient data real-world data (IPD-RWD) and to compare these methods with recommendations made in guidelines from European regulatory and health technology assessment (HTA) organizations and with their evaluations described in assessment reports.</p><p><strong>Methods: </strong>A systematic literature review (until March 1, 2023) in PubMed and Connected Papers was performed to identify analytical methods for comparing uncontrolled trials with external controls from IPD-RWD. These methods were compared descriptively with methods recommended in method guidelines and encountered in assessment reports of the European Medicines Agency (2015-2020) and 4 European HTA organizations (2015-2023).</p><p><strong>Results: </strong>Thirty-four identified scientific articles described analytical methods for comparing uncontrolled trial data with IPD-RWD-based external controls. The various methods covered controlling for confounding and/or dependent censoring, correction for missing data, and analytical comparative modeling methods. Seven guidelines also focused on research design, RWD quality, and transparency aspects, and 4 of those recommended analytical methods for comparisons with IPD-RWD. The methods discussed in regulatory (n = 15) and HTA (n = 35) assessment reports were often based on aggregate data and lacked transparency owing to the few details provided.</p><p><strong>Conclusions: </strong>Literature and guidelines suggest a methodological approach to comparing uncontrolled trials with external controls from IPD-RWD similar to target trial emulation, using state-of-the-art methods. External controls supporting regulatory and HTA decision making were rarely in line with this approach. Twelve recommendations are proposed to improve the quality and acceptability of these methods.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142146419","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Integrating Price Benchmarks and Comparative Clinical Effectiveness to Inform the Medicare Drug Price Negotiation Program 整合价格基准和临床效果比较,为医疗保险药品价格谈判计划提供信息。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-21 DOI: 10.1016/j.jval.2024.08.001
Sean D. Sullivan BScPharm, PhD , Olivier J. Wouters PhD , Emma M. Cousin PharmD , Ayuri S. Kirihennedige BSc , Inmaculada Hernandez PharmD, PhD

Objectives

By September 2024, the Centers for Medicare and Medicaid Services (CMS) will publicly report the negotiated prices (Maximum Fair Prices) for the first 10 drugs selected for price negotiation. We estimate initial price offers based on net prices, statutorily defined ceilings, and comparative effectiveness data for the 10 drugs and their therapeutic alternatives.

Methods

We utilized net prices and other price benchmarks for the 10 drugs and their therapeutic alternatives. We searched for data on comparative clinical effectiveness for the primary indications. We outlined a range of plausible initial price offers based on CMS guidance and our interpretation of regulatory intent.

Results

For ibrutinib and ustekinumab, statutorily defined ceiling prices will likely determine the initial price offers. The integration of net pricing and clinical evidence from comparator branded products will inform the initial price offers for apixaban, empagliflozin, etanercept, and insulin aspart. Rivaroxaban and sacubitril/valsartan have therapeutic alternatives that are generics; therefore, CMS may apply a discount to current net prices. To achieve savings in the negotiation of dapagliflozin and sitagliptin, CMS will have to leverage additional negotiation factors because statutory defined ceilings and net prices of therapeutic alternatives are similar or higher.

Conclusions

This analysis sheds light on important price benchmarks and clinical evidence factors for the determination of the initial price offers. Although we were not able to simulate the offer and counter-offer process, our findings provide a transparent and systematic way to produce initial offers that are consistent with CMS guidance.
目标:到 2024 年 9 月,医疗保险和医疗补助服务中心(CMS)将公开报告首批选定进行价格谈判的十种药物的谈判价格(最高公平价格)。我们根据这十种药物及其替代治疗药物的净价、法定最高限价和比较效果数据估算了初始报价:我们利用了这十种药物及其替代治疗药物的净价格和其他价格基准。我们搜索了主要适应症的临床疗效比较数据。根据 CMS 指南和我们对监管意图的理解,我们列出了一系列合理的初始报价:对于伊布替尼和乌司替尼,法定的最高限价可能会决定初始报价。阿哌沙班、恩格列净、依那西普和阿斯巴甜胰岛素的初始报价将参考参照品牌产品的净定价和临床证据。利伐沙班和sacubitril/valsartan的治疗替代品是仿制药,因此CMS可能会在当前净价的基础上打折。为了在达帕利洛嗪和西他列汀的谈判中实现节约,CMS 将不得不利用更多的谈判因素,因为法定定义的上限和治疗替代品的净价格相似或更高:本分析揭示了确定初始报价的重要价格基准和临床证据因素。虽然我们无法模拟报价和还价过程,但我们的研究结果为制定初始报价提供了一种透明、系统的方法。
{"title":"Integrating Price Benchmarks and Comparative Clinical Effectiveness to Inform the Medicare Drug Price Negotiation Program","authors":"Sean D. Sullivan BScPharm, PhD ,&nbsp;Olivier J. Wouters PhD ,&nbsp;Emma M. Cousin PharmD ,&nbsp;Ayuri S. Kirihennedige BSc ,&nbsp;Inmaculada Hernandez PharmD, PhD","doi":"10.1016/j.jval.2024.08.001","DOIUrl":"10.1016/j.jval.2024.08.001","url":null,"abstract":"<div><h3>Objectives</h3><div>By September 2024, the Centers for Medicare and Medicaid Services (CMS) will publicly report the negotiated prices (Maximum Fair Prices) for the first 10 drugs selected for price negotiation. We estimate initial price offers based on net prices, statutorily defined ceilings, and comparative effectiveness data for the 10 drugs and their therapeutic alternatives.</div></div><div><h3>Methods</h3><div>We utilized net prices and other price benchmarks for the 10 drugs and their therapeutic alternatives. We searched for data on comparative clinical effectiveness for the primary indications. We outlined a range of plausible initial price offers based on CMS guidance and our interpretation of regulatory intent.</div></div><div><h3>Results</h3><div>For ibrutinib and ustekinumab, statutorily defined ceiling prices will likely determine the initial price offers. The integration of net pricing and clinical evidence from comparator branded products will inform the initial price offers for apixaban, empagliflozin, etanercept, and insulin aspart. Rivaroxaban and sacubitril/valsartan have therapeutic alternatives that are generics; therefore, CMS may apply a discount to current net prices. To achieve savings in the negotiation of dapagliflozin and sitagliptin, CMS will have to leverage additional negotiation factors because statutory defined ceilings and net prices of therapeutic alternatives are similar or higher.</div></div><div><h3>Conclusions</h3><div>This analysis sheds light on important price benchmarks and clinical evidence factors for the determination of the initial price offers. Although we were not able to simulate the offer and counter-offer process, our findings provide a transparent and systematic way to produce initial offers that are consistent with CMS guidance.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"27 10","pages":"Pages 1348-1357"},"PeriodicalIF":4.9,"publicationDate":"2024-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142000750","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The Impact of Additive Population(s), Intervention, Comparator(s), and Outcomes in a European Joint Clinical Health Technology Assessment. 欧洲联合临床健康技术评估中附加 PICOs 的影响。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-10 DOI: 10.1016/j.jval.2024.07.024
Anke van Engen, Robert Krüger, Adam Parnaby, Mihai Rotaru, James Ryan, Dima Samaha, Dimitrios Tzelis

Objectives: To assess the potential number of European Union (EU) population(s), intervention, comparator(s), and outcomes (PICOs) based on European Network for Health Technology Assessment 21 (EUnetHTA 21) guidance and to explore further evidence-based opportunities to produce more predictable and workable EU PICOs.

Methods: The consolidated EU PICOs of 2 future hypothetical medicines in first-line non-small cell lung cancer (1L NSCLC) and third line multiple myeloma (3L MM) were derived using published health technology assessment reports of 2 recent medicines in similar indications based on EUnetHTA 21 proposed guidance. Sensitivity analysis assessed the impact of additional PICO requests. The number of analyses requested was estimated.

Results: In 1L NSCLC and 3L MM, 6 and 9 EU Member States (MS), respectively, had published health technology assessment reports. PICO consolidation resulted in 10 PICOs for 1L NSCLC and 16 PICOs for 3L MM, increasing to 14 and 18 PICOs, respectively, when England's National Institute for Health and Care Excellence scope was included to proxy remaining MS. A minimum of 280 and 720 analyses would be requested, exponentially increasing as additional outcome measures and subgroups are requested.

Conclusions: The PICO approach outlined by EUnetHTA 21 results in a significant number of analysis requests and substantial resources. Use of complementary analyses alongside evidence-based methods to derive PICOs and engaging with the health technology developer throughout the process would create a workable EU PICO that is predictable and most impactful for the EU, resulting in a timely and high-quality assessment report that is more usable at a MS level.

目的评估基于 EUnetHTA 21 指南的欧盟 PICO 的潜在数量,并探索更多基于证据的机会,以产生更可预测、更可行的欧盟 PICO:方法:根据 EUnetHTA 21 提出的指南,利用最近两种类似适应症药物的 HTA 公开报告,得出了两种未来假定药物在一线非小细胞肺癌 (1L NSCLC) 和三线多发性骨髓瘤 (3L MM) 中的综合欧盟 PICO。敏感性分析评估了额外 PICO 请求的影响。对要求进行的分析数量进行了估算:对于 1L NSCLC 和 3L MM,分别有 6 个和 9 个欧盟成员国 (MS) 发布了 HTA 报告。PICO 合并后,1L NSCLC 和 3L MM 分别有 10 个和 16 个 PICO,如果将英格兰的 NICE 范围纳入其余 MS 的代理范围,则分别增加到 14 个和 18 个 PICO。至少需要进行 280 项和 720 项分析,随着要求增加结果测量和亚组,分析数量将呈指数增长:结论:EUnetHTA 21 概述的 PICO 方法会导致大量的分析请求和大量的资源。使用补充分析和循证方法来推导 PICO,并在整个过程中与卫生技术开发者合作,将创建一个可行的、可预测的、对欧盟影响最大的欧盟 PICO,从而产生一份及时的、高质量的评估报告,在成员国层面上更易于使用。
{"title":"The Impact of Additive Population(s), Intervention, Comparator(s), and Outcomes in a European Joint Clinical Health Technology Assessment.","authors":"Anke van Engen, Robert Krüger, Adam Parnaby, Mihai Rotaru, James Ryan, Dima Samaha, Dimitrios Tzelis","doi":"10.1016/j.jval.2024.07.024","DOIUrl":"10.1016/j.jval.2024.07.024","url":null,"abstract":"<p><strong>Objectives: </strong>To assess the potential number of European Union (EU) population(s), intervention, comparator(s), and outcomes (PICOs) based on European Network for Health Technology Assessment 21 (EUnetHTA 21) guidance and to explore further evidence-based opportunities to produce more predictable and workable EU PICOs.</p><p><strong>Methods: </strong>The consolidated EU PICOs of 2 future hypothetical medicines in first-line non-small cell lung cancer (1L NSCLC) and third line multiple myeloma (3L MM) were derived using published health technology assessment reports of 2 recent medicines in similar indications based on EUnetHTA 21 proposed guidance. Sensitivity analysis assessed the impact of additional PICO requests. The number of analyses requested was estimated.</p><p><strong>Results: </strong>In 1L NSCLC and 3L MM, 6 and 9 EU Member States (MS), respectively, had published health technology assessment reports. PICO consolidation resulted in 10 PICOs for 1L NSCLC and 16 PICOs for 3L MM, increasing to 14 and 18 PICOs, respectively, when England's National Institute for Health and Care Excellence scope was included to proxy remaining MS. A minimum of 280 and 720 analyses would be requested, exponentially increasing as additional outcome measures and subgroups are requested.</p><p><strong>Conclusions: </strong>The PICO approach outlined by EUnetHTA 21 results in a significant number of analysis requests and substantial resources. Use of complementary analyses alongside evidence-based methods to derive PICOs and engaging with the health technology developer throughout the process would create a workable EU PICO that is predictable and most impactful for the EU, resulting in a timely and high-quality assessment report that is more usable at a MS level.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-08-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141917572","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Age and Gender Differences in the Relationship Between Chronic Pain and Dementia Among Older Australians. 澳大利亚老年人慢性疼痛与痴呆症之间关系的年龄和性别差异。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-09 DOI: 10.1016/j.jval.2024.07.022
Rezwanul Haque, Khorshed Alam, Jeff Gow, Christine Neville, Syed Afroz Keramat

Objectives: Chronic pain is a highly debilitating condition that affects older adults and has the potential to increase their odds of experiencing cognitive impairment. The primary objective of this study was to examine the correlation between chronic pain and dementia. Additionally, this research endeavors to ascertain whether the association between chronic pain and dementia differs by age and gender.

Methods: Cross-sectional data were derived from the Survey of Disability, Ageing, and Carers. A total of 20 671 and 20 081 participants aged 65 years and older in 2015 and 2018, respectively, were included in this study. The pooled association between chronic pain and dementia was assessed using a multivariable logistic regression model. Furthermore, the study also examined the multiplicative interaction effects between chronic pain and age, as well as chronic pain and gender, with dementia.

Results: The pooled analysis demonstrated that chronic pain was associated with a heightened odds of dementia (adjusted odds ratio 1.95; 95% CI 1.85-2.05) among older Australians compared with their counterparts without chronic pain. The interaction effect indicated that individuals with chronic pain across all age groups exhibited increased odds of living with dementia. Additionally, women with chronic pain had higher odds of dementia compared with their counterparts without chronic pain and being male.

Conclusions: A continuous, coordinated, and tailored healthcare strategy is necessary to determine the pain management goals and explore early treatment options for chronic pain in older adults, particularly in groups with the greatest need.

目的:慢性疼痛是一种对老年人影响极大的衰弱性疾病,有可能增加老年人出现认知障碍的几率。本研究的主要目的是探讨慢性疼痛与痴呆症之间的相关性。此外,本研究还试图确定慢性疼痛与痴呆症之间的关联是否因年龄和性别而异:横断面数据来自于残疾、老龄化和照顾者调查(SDAC)。本研究分别纳入了2015年和2018年65岁及以上的20671名和20081名参与者。采用多变量逻辑回归模型评估了慢性疼痛与痴呆症之间的集合关联。此外,研究还考察了慢性疼痛与年龄、慢性疼痛与性别与痴呆之间的乘法交互效应:汇总分析表明,与无慢性疼痛的澳大利亚老年人相比,慢性疼痛与痴呆症的发生几率增加有关(调整后患病率比 [AOR]=1.95, 95%CI: 1.85-2.05)。交互效应表明,所有年龄组的慢性疼痛患者患痴呆症的几率都有所增加。此外,与无慢性疼痛的女性和男性相比,患有慢性疼痛的女性患痴呆症的几率更高:有必要采取持续、协调和量身定制的医疗保健策略,以确定疼痛管理目标并探索老年人慢性疼痛的早期治疗方案,尤其是在需求最大的群体中。
{"title":"Age and Gender Differences in the Relationship Between Chronic Pain and Dementia Among Older Australians.","authors":"Rezwanul Haque, Khorshed Alam, Jeff Gow, Christine Neville, Syed Afroz Keramat","doi":"10.1016/j.jval.2024.07.022","DOIUrl":"10.1016/j.jval.2024.07.022","url":null,"abstract":"<p><strong>Objectives: </strong>Chronic pain is a highly debilitating condition that affects older adults and has the potential to increase their odds of experiencing cognitive impairment. The primary objective of this study was to examine the correlation between chronic pain and dementia. Additionally, this research endeavors to ascertain whether the association between chronic pain and dementia differs by age and gender.</p><p><strong>Methods: </strong>Cross-sectional data were derived from the Survey of Disability, Ageing, and Carers. A total of 20 671 and 20 081 participants aged 65 years and older in 2015 and 2018, respectively, were included in this study. The pooled association between chronic pain and dementia was assessed using a multivariable logistic regression model. Furthermore, the study also examined the multiplicative interaction effects between chronic pain and age, as well as chronic pain and gender, with dementia.</p><p><strong>Results: </strong>The pooled analysis demonstrated that chronic pain was associated with a heightened odds of dementia (adjusted odds ratio 1.95; 95% CI 1.85-2.05) among older Australians compared with their counterparts without chronic pain. The interaction effect indicated that individuals with chronic pain across all age groups exhibited increased odds of living with dementia. Additionally, women with chronic pain had higher odds of dementia compared with their counterparts without chronic pain and being male.</p><p><strong>Conclusions: </strong>A continuous, coordinated, and tailored healthcare strategy is necessary to determine the pain management goals and explore early treatment options for chronic pain in older adults, particularly in groups with the greatest need.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141914152","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cost-Effectiveness Analysis of Bevacizumab Biosimilars Versus Originator Bevacizumab for Metastatic Colorectal Cancer: A Comparative Study Using Real-World Data. 贝伐珠单抗生物仿制药与原研贝伐珠单抗治疗转移性结直肠癌的成本效益分析:一项使用真实世界数据的比较研究。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-09 DOI: 10.1016/j.jval.2024.07.018
Brandon Lu, Erind Dvorani, Lena Nguyen, Jaclyn M Beca, Rebecca E Mercer, Andrea Adamic, Caroline Muñoz, Kelvin K W Chan

Objectives: MVASI (Amgen) and Zirabev (Pfizer) are 2 of the earliest bevacizumab biosimilars approved for the first-line treatment of metastatic colorectal cancer (mCRC). We aimed to confirm and quantify the real-world cost savings and cost-effectiveness of MVASI and Zirabev relative to originator bevacizumab for patients with mCRC.

Methods: We conducted a population-based, retrospective cohort study in Ontario, Canada, where originator and biosimilar bevacizumab are universally publicly funded. All mCRC patients who received originator bevacizumab between January 2008 and August 2019 or biosimilar bevacizumab between August 2019 and March 2021 were propensity score matched (1:4) to adjust for baseline differences. Total 1-year patient-level costs (CAD) and effects (life years [LY] and quality-adjusted LYs) were calculated from the public health payer's perspective. Primary outcomes included incremental net monetary benefit and incremental net health benefit (INHB). Sensitivity analyses included a subgroup analysis by biosimilar type (MVASI/Zirabev) and a 2-year analysis.

Results: The matched cohort included 747 biosimilar cases and 2945 comparators. Bevacizumab biosimilars were associated with an incremental cost of -$6379 (95%CI: -9417, -3537) (ie, cost saving) and incremental effect of 0.0 (95% CI: -0.02, 0.02) LY and -0.01 (95% CI: -0.03, 0) quality-adjusted LYs gained. Incremental net monetary benefit and INHB estimates were $6331 (95% CI: 6245, 6417) and 0.127 LY (95% CI: 0.125, 0.128), respectively, at a willingness-to-pay threshold of $50 000/life year gained, with all estimates indicating the cost-effectiveness of biosimilar bevacizumab. Cost-effectiveness remained consistent across biosimilar brand subgroups and 2-year sensitivity analyses.

Conclusion: Bevacizumab biosimilars demonstrated real-world cost savings while providing similar survival benefit as originator bevacizumab, confirming the initial expectations of their implementation and supporting health system sustainability.

目的:MVASI(安进公司)和Zirabev(辉瑞公司)是最早获准用于转移性结直肠癌(mCRC)一线治疗的两种贝伐珠单抗生物仿制药。我们的目的是确认和量化 MVASI 和 Zirabev 相对于原研贝伐珠单抗治疗 mCRC 患者的实际成本节约和成本效益:我们在加拿大安大略省开展了一项基于人群的回顾性队列研究,该省的原研药和生物仿制药贝伐珠单抗均由政府资助。对 2008 年 1 月至 2019 年 8 月期间接受原研贝伐珠单抗治疗或 2019 年 8 月至 2021 年 3 月期间接受生物类似物贝伐珠单抗治疗的所有 mCRC 患者进行倾向评分匹配(1:4),以调整基线差异。从公共卫生支付方的角度计算了1年患者层面的总成本(CAD)和效果(生命年(LY)和质量调整生命年(QALY))。主要结果包括增量净货币效益(INMB)和增量净健康效益(INHB)。敏感性分析包括按生物类似药类型(MVASI/Zirabev)进行的亚组分析和两年分析:匹配队列包括 747 个生物类似药病例和 2,945 个比较者。贝伐珠单抗生物仿制药的增量成本为-6,379美元(95%CI:-9,417, -3,537)(即节省成本),增量效果为0.0(95%CI:-0.02, 0.02)LY和-0.01(95%CI:-0.03, 0)QALY。在 50,000 美元/LYG 的支付意愿阈值下,INMB 和 INHB 的估计值分别为 6,331 美元(95% CI:6,245, 6,417)和 0.127 LY(95% CI:0.125, 0.128),所有估计值均表明生物类似药贝伐珠单抗具有成本效益。在生物类似药品牌亚组和2年敏感性分析中,成本效益保持一致:贝伐珠单抗生物仿制药在提供与原研药贝伐珠单抗相似的生存获益的同时,还显示出了实际的成本节约效果,证实了最初对其实施的预期,并支持了医疗系统的可持续性。
{"title":"Cost-Effectiveness Analysis of Bevacizumab Biosimilars Versus Originator Bevacizumab for Metastatic Colorectal Cancer: A Comparative Study Using Real-World Data.","authors":"Brandon Lu, Erind Dvorani, Lena Nguyen, Jaclyn M Beca, Rebecca E Mercer, Andrea Adamic, Caroline Muñoz, Kelvin K W Chan","doi":"10.1016/j.jval.2024.07.018","DOIUrl":"10.1016/j.jval.2024.07.018","url":null,"abstract":"<p><strong>Objectives: </strong>MVASI (Amgen) and Zirabev (Pfizer) are 2 of the earliest bevacizumab biosimilars approved for the first-line treatment of metastatic colorectal cancer (mCRC). We aimed to confirm and quantify the real-world cost savings and cost-effectiveness of MVASI and Zirabev relative to originator bevacizumab for patients with mCRC.</p><p><strong>Methods: </strong>We conducted a population-based, retrospective cohort study in Ontario, Canada, where originator and biosimilar bevacizumab are universally publicly funded. All mCRC patients who received originator bevacizumab between January 2008 and August 2019 or biosimilar bevacizumab between August 2019 and March 2021 were propensity score matched (1:4) to adjust for baseline differences. Total 1-year patient-level costs (CAD) and effects (life years [LY] and quality-adjusted LYs) were calculated from the public health payer's perspective. Primary outcomes included incremental net monetary benefit and incremental net health benefit (INHB). Sensitivity analyses included a subgroup analysis by biosimilar type (MVASI/Zirabev) and a 2-year analysis.</p><p><strong>Results: </strong>The matched cohort included 747 biosimilar cases and 2945 comparators. Bevacizumab biosimilars were associated with an incremental cost of -$6379 (95%CI: -9417, -3537) (ie, cost saving) and incremental effect of 0.0 (95% CI: -0.02, 0.02) LY and -0.01 (95% CI: -0.03, 0) quality-adjusted LYs gained. Incremental net monetary benefit and INHB estimates were $6331 (95% CI: 6245, 6417) and 0.127 LY (95% CI: 0.125, 0.128), respectively, at a willingness-to-pay threshold of $50 000/life year gained, with all estimates indicating the cost-effectiveness of biosimilar bevacizumab. Cost-effectiveness remained consistent across biosimilar brand subgroups and 2-year sensitivity analyses.</p><p><strong>Conclusion: </strong>Bevacizumab biosimilars demonstrated real-world cost savings while providing similar survival benefit as originator bevacizumab, confirming the initial expectations of their implementation and supporting health system sustainability.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141914153","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Proposal for a General Outcome-Based Value Attribution Framework for Combination Therapies. 关于基于成果的组合疗法价值归属总体框架的建议。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-09 DOI: 10.1016/j.jval.2024.07.019
Lotte Steuten, Mickael Lothgren, Andrew Bruce, Marco Campioni, Adrian Towse

Objectives: Valuing and pricing the components of combination therapies can be difficult because of competition law issues, difficulty implementing different prices for the same product in alternative uses, and attributing value to each component of the combination. We propose a value attribution solution that allows all combination components to be priced according to their relative value in the combination.

Methods: We developed a value attribution solution that is universal, symmetrical, and neutral to each combination constituent, regardless of whether it is the backbone or the add-on, and complete, meaning that it will always attribute the full value of the combination between the component parts. Moreover, it can be applied to any number of components in the combination (eg, triplets or quadruplets). We compared this solution with 2 other existing approaches.

Results: The results of the proposed value attribution solution sit between those of the 2 other value attribution approaches as it combines elements of each. As the degree of additivity moves further away from one in either direction, then our general approach ratios also move, reflecting the impact of the incremental value.

Conclusions: The proposed value attribution solution for combination therapies differs from 2 existing approaches by being universally applicable and allowing for symmetry when neutral to the constituent components of the combination. To optimally contribute to policy debate and practice, various requirements for its implementation need to be well understood, including how to overcome (1) partial information, (2) whether its assumptions can be relaxed, and (3) implementation issues.

目的:由于竞争法问题、同一产品在不同用途中执行不同价格的困难,以及对组合疗法各组成部分的价值归属问题,对组合疗法各组成部分进行估值和定价可能很困难。我们提出了一种价值归属解决方案,可以根据组合疗法中所有组合成分的相对价值对其进行定价:方法:我们开发了一种价值归属解决方案,它具有通用性、对称性,并且对每个组合成分都是中性的,无论其是主干还是附加成分;它还具有完整性,这意味着它总是在各组成部分之间归属组合的全部价值。此外,它还适用于组合中任何数量的成分(如三胞胎或四胞胎)。我们将这一解决方案与其他两种现有方法进行了比较:结果:建议的价值归属解决方案的结果介于其他两种价值归属方法之间,因为它结合了每种方法的元素。随着可加性程度在任一方向上进一步偏离 1,我们的一般方法比率也会发生变化,从而反映出增量价值的影响:针对组合疗法提出的价值归因解决方案不同于现有的两种方法,它具有普遍适用性,并允许在对组合疗法的组成成分保持中立时实现对称。为了对政策辩论和实践做出最佳贡献,需要充分了解其实施的各种要求,包括如何克服(1)信息不全面,(2)是否可以放宽其假设,以及(3)实施问题。
{"title":"Proposal for a General Outcome-Based Value Attribution Framework for Combination Therapies.","authors":"Lotte Steuten, Mickael Lothgren, Andrew Bruce, Marco Campioni, Adrian Towse","doi":"10.1016/j.jval.2024.07.019","DOIUrl":"10.1016/j.jval.2024.07.019","url":null,"abstract":"<p><strong>Objectives: </strong>Valuing and pricing the components of combination therapies can be difficult because of competition law issues, difficulty implementing different prices for the same product in alternative uses, and attributing value to each component of the combination. We propose a value attribution solution that allows all combination components to be priced according to their relative value in the combination.</p><p><strong>Methods: </strong>We developed a value attribution solution that is universal, symmetrical, and neutral to each combination constituent, regardless of whether it is the backbone or the add-on, and complete, meaning that it will always attribute the full value of the combination between the component parts. Moreover, it can be applied to any number of components in the combination (eg, triplets or quadruplets). We compared this solution with 2 other existing approaches.</p><p><strong>Results: </strong>The results of the proposed value attribution solution sit between those of the 2 other value attribution approaches as it combines elements of each. As the degree of additivity moves further away from one in either direction, then our general approach ratios also move, reflecting the impact of the incremental value.</p><p><strong>Conclusions: </strong>The proposed value attribution solution for combination therapies differs from 2 existing approaches by being universally applicable and allowing for symmetry when neutral to the constituent components of the combination. To optimally contribute to policy debate and practice, various requirements for its implementation need to be well understood, including how to overcome (1) partial information, (2) whether its assumptions can be relaxed, and (3) implementation issues.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141914159","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cost-of/Burden-of-Illness Studies: Steps Backward? 疾病成本/负担研究:退步?
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-09 DOI: 10.1016/j.jval.2024.07.023
Brian E. Rittenhouse PhD
{"title":"Cost-of/Burden-of-Illness Studies: Steps Backward?","authors":"Brian E. Rittenhouse PhD","doi":"10.1016/j.jval.2024.07.023","DOIUrl":"10.1016/j.jval.2024.07.023","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"27 10","pages":"Pages 1466-1467"},"PeriodicalIF":4.9,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141914155","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cost-Utility Analysis of Antipsychotic Reduction and Discontinuation in Patients With Long-Term Schizophrenia and Psychosis in English Mental Health Trusts: The RADAR Study. 英国精神卫生信托基金中长期精神分裂症和精神病患者减少和停用抗精神病药物的成本效用分析:RADAR 研究》。
IF 4.9 2区 医学 Q1 ECONOMICS Pub Date : 2024-08-09 DOI: 10.1016/j.jval.2024.07.017
George Bray, Joanna Moncrieff, Stefan Priebe, Louise Marston, Glyn Lewis, Nadia Haynes, Vanessa Pinfold, Sonia Johnson, Rachael Maree Hunter

Objectives: The current recommended treatment for patients with recurrent episodes of schizophrenia and related conditions is antipsychotic medication. However, many antipsychotic users remain functionally impaired and experience serious physical and mental side effects. This study aims to assess the cost-effectiveness of a gradual antipsychotic reduction and discontinuation strategy compared with maintenance treatment over 24 months from mental health services, health and social care, and societal perspectives.

Methods: Nineteen mental health trusts recruited patients to the Research into Antipsychotic Discontinuation and Reduction (RADAR) randomized controlled trial. Quality-adjusted life-years were calculated from patient-reported EQ-5D-5L, with years of full capability calculated from the patient-reported ICECAP-A. Mental health services use and medication was collected from medical records. Other resource use and productivity loss was collected using self-completed questionnaires. Costs were calculated from published sources.

Results: A total of 253 participants were randomized: 126 assigned to antipsychotic dose reduction and 127 to maintenance. There were no significant differences between arms in total costs for any perspectives. There were no significant difference in quality-adjusted life-years (-0.035; 95% CI: -0.123 to 0.052), whereas years of full capability were significantly lower in the reduction arm compared with the maintenance arm (baseline-adjusted difference: -0.103; 95% CI: -0.192 to -0.014). The reduction strategy was dominated by maintenance for all analyses and was not likely to be cost-effective.

Conclusions: It is unlikely that gradual antipsychotic reduction and discontinuation strategy is cost-effective compared with maintenance over 2-years for patients with schizophrenia and other recurrent psychotic disorders who are on long-term antipsychotics.

目的:对于反复发作的精神分裂症及相关疾病患者,目前推荐的治疗方法是服用抗精神病药物。然而,许多服用抗精神病药物的患者仍然功能受损,并出现严重的身体和精神副作用。本研究旨在从精神卫生服务、卫生和社会保健以及社会角度评估逐步减少和停用抗精神病药物的策略与维持治疗 24 个月的成本效益:19家精神卫生信托机构招募患者参加RADAR随机对照试验。质量调整生命年(QALYs)根据患者报告的EQ-5D-5L计算,完全能力年(YFCs)根据患者报告的ICECAP-A计算。心理健康服务的使用和用药情况由医疗记录收集。其他资源使用和生产力损失通过自填问卷收集。结果:253名参与者被随机分配:126人被分配到减少抗精神病药物剂量组,127人被分配到维持治疗组。两组患者在任何方面的总费用均无明显差异。QALYs方面无明显差异(-0.035;95% CI:-0.123至0.052),而减量治疗组的YFCs明显低于维持治疗组(基线调整后差异:-0.103;95% CI:-0.192至-0.014)。在所有分析中,减量治疗策略都被维持治疗策略所取代,因此不太可能具有成本效益:结论:对于长期服用抗精神病药物的精神分裂症和其他复发性精神障碍患者而言,与维持治疗两年相比,逐步减少和停用抗精神病药物的策略不太可能具有成本效益。
{"title":"Cost-Utility Analysis of Antipsychotic Reduction and Discontinuation in Patients With Long-Term Schizophrenia and Psychosis in English Mental Health Trusts: The RADAR Study.","authors":"George Bray, Joanna Moncrieff, Stefan Priebe, Louise Marston, Glyn Lewis, Nadia Haynes, Vanessa Pinfold, Sonia Johnson, Rachael Maree Hunter","doi":"10.1016/j.jval.2024.07.017","DOIUrl":"10.1016/j.jval.2024.07.017","url":null,"abstract":"<p><strong>Objectives: </strong>The current recommended treatment for patients with recurrent episodes of schizophrenia and related conditions is antipsychotic medication. However, many antipsychotic users remain functionally impaired and experience serious physical and mental side effects. This study aims to assess the cost-effectiveness of a gradual antipsychotic reduction and discontinuation strategy compared with maintenance treatment over 24 months from mental health services, health and social care, and societal perspectives.</p><p><strong>Methods: </strong>Nineteen mental health trusts recruited patients to the Research into Antipsychotic Discontinuation and Reduction (RADAR) randomized controlled trial. Quality-adjusted life-years were calculated from patient-reported EQ-5D-5L, with years of full capability calculated from the patient-reported ICECAP-A. Mental health services use and medication was collected from medical records. Other resource use and productivity loss was collected using self-completed questionnaires. Costs were calculated from published sources.</p><p><strong>Results: </strong>A total of 253 participants were randomized: 126 assigned to antipsychotic dose reduction and 127 to maintenance. There were no significant differences between arms in total costs for any perspectives. There were no significant difference in quality-adjusted life-years (-0.035; 95% CI: -0.123 to 0.052), whereas years of full capability were significantly lower in the reduction arm compared with the maintenance arm (baseline-adjusted difference: -0.103; 95% CI: -0.192 to -0.014). The reduction strategy was dominated by maintenance for all analyses and was not likely to be cost-effective.</p><p><strong>Conclusions: </strong>It is unlikely that gradual antipsychotic reduction and discontinuation strategy is cost-effective compared with maintenance over 2-years for patients with schizophrenia and other recurrent psychotic disorders who are on long-term antipsychotics.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141914156","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
期刊
Value in Health
全部 Acc. Chem. Res. ACS Applied Bio Materials ACS Appl. Electron. Mater. ACS Appl. Energy Mater. ACS Appl. Mater. Interfaces ACS Appl. Nano Mater. ACS Appl. Polym. Mater. ACS BIOMATER-SCI ENG ACS Catal. ACS Cent. Sci. ACS Chem. Biol. ACS Chemical Health & Safety ACS Chem. Neurosci. ACS Comb. Sci. ACS Earth Space Chem. ACS Energy Lett. ACS Infect. Dis. ACS Macro Lett. ACS Mater. Lett. ACS Med. Chem. Lett. ACS Nano ACS Omega ACS Photonics ACS Sens. ACS Sustainable Chem. Eng. ACS Synth. Biol. Anal. Chem. BIOCHEMISTRY-US Bioconjugate Chem. BIOMACROMOLECULES Chem. Res. Toxicol. Chem. Rev. Chem. Mater. CRYST GROWTH DES ENERG FUEL Environ. Sci. Technol. Environ. Sci. Technol. Lett. Eur. J. Inorg. Chem. IND ENG CHEM RES Inorg. Chem. J. Agric. Food. Chem. J. Chem. Eng. Data J. Chem. Educ. J. Chem. Inf. Model. J. Chem. Theory Comput. J. Med. Chem. J. Nat. Prod. J PROTEOME RES J. Am. Chem. Soc. LANGMUIR MACROMOLECULES Mol. Pharmaceutics Nano Lett. Org. Lett. ORG PROCESS RES DEV ORGANOMETALLICS J. Org. Chem. J. Phys. Chem. J. Phys. Chem. A J. Phys. Chem. B J. Phys. Chem. C J. Phys. Chem. Lett. Analyst Anal. Methods Biomater. Sci. Catal. Sci. Technol. Chem. Commun. Chem. Soc. Rev. CHEM EDUC RES PRACT CRYSTENGCOMM Dalton Trans. Energy Environ. Sci. ENVIRON SCI-NANO ENVIRON SCI-PROC IMP ENVIRON SCI-WAT RES Faraday Discuss. Food Funct. Green Chem. Inorg. Chem. Front. Integr. Biol. J. Anal. At. Spectrom. J. Mater. Chem. A J. Mater. Chem. B J. Mater. Chem. C Lab Chip Mater. Chem. Front. Mater. Horiz. MEDCHEMCOMM Metallomics Mol. Biosyst. Mol. Syst. Des. Eng. Nanoscale Nanoscale Horiz. Nat. Prod. Rep. New J. Chem. Org. Biomol. Chem. Org. Chem. Front. PHOTOCH PHOTOBIO SCI PCCP Polym. Chem.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1